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Vertex's Cystic Fibrosis Combo Gets Priority Review in U.S.

Vertex Pharmaceuticals Incorporated (VRTX - Free Report) announced that its regulatory applications for tezacaftor/ivacaftor (Kalydeco) combination have been accepted by the FDA and the European Medicines Agency (EMA) for review.

The regulatory applications have been filed to seek approval of Kalydeco in combination with tezacaftor (VX-661) for the treatment of cystic fibrosis (CF) patients (12 and older) with two copies of the F508del mutation.

We note that Kalydeco is already approved for the treatment of patients (two years and above) with CF who have one of 33 mutations in their cystic fibrosis transmembrane conductance regulator (CFTR) gene.

Meanwhile, the application in the U.S. has been granted a priority review with the FDA expected to give its decision on Feb 28, 2018.

Shares of Vertex were up 1.9% on Thursday. In-fact, Vertex’s share price is up a massive 111.9% so far this year, comparing favorably with an increase of 7.9%for the industry.

Coming back to the latest news, the regulatory applications in the U.S. and EU were submitted on the basis of positive outcome from two phase III studies – EVOLVE and EXPAND. Data from the studies was announced in March this year. Both studies met their primary endpoints and demonstrated statistically significant improvements in lung function in patients with CF.

The combination had also been granted Orphan Drug designation by the FDA.

Per the company’s press release, approximately 75,000 people in North America, Europe and Australia suffer from CF. In fact, more than 29% people of ages 12 years or older have two copies of the F508del mutation, and more than 1,500 people of the age 12 have one mutation. Hence, approval of the drug will provide the company access to a huge base of patients affected by the disease and bring it closer to the goal of serving people with the disease.

In fact, Vertex’s CF pipeline is quite strong with a broad portfolio of next-generation CF correctors.

Vertex is evaluating some next-generation CFTR correctors (VX-152, VX-440, VX-659 and VX-445) as part of a triple combination with tezacaftor and ivacaftor.

Data from VX-152 and VX-440 phase II and VX-659 phase I triple combination studies, conducted on CF patients who have one F508del mutation and one minimal function mutation (F508del/Min), were presented in July 2017. The data demonstrated that all three combinations led to pronounced improvement in lung function. In fact, these are the first data to show the potential to treat the underlying cause of CF in patients who have a severe and difficult-to-treat type of the disease.

Following discussions with regulatory agencies, Vertex will initiate pivotal phase III studies on one or two of the four triple combination regimens in the first half of 2018.

Its triple combination CF regimens are considered crucial for long-term growth. If the triple-combo regimes are successful, Vertex can address a significantly larger CF patient population– almost 90% of patients with CF - in the future.

Shares of Alexion are up 12.7% year to date while estimates for both 2017 and 2018 have risen by 5.5% over the past 30 days.

Shares of Ligand have risen 24.8% so far this year while estimates for 2017 and 2018 have risen by 10.4% and 2.2%, respectively, over the past 30 days.

Regeneron’s shares up 32.2% this year so far. Estimates have risen 15.1% for 2017 while that for 2018 have gone up by 5.8% over the past 30 days.

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